What is the prognosis of Langerhans cell histiocytosis (LCH)?

Updated: Jun 12, 2020
  • Author: Christopher R Shea, MD; Chief Editor: William D James, MD  more...
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Answer

More than half the patients younger than 2 years with disseminated Langerhans cell histiocytosis (LCH) and organ dysfunction die of the disease, whereas unifocal LCH and most cases of congenital self-healing histiocytosis are self-limited. Multifocal chronic LCH is self-limited in most cases, but increased mortality has been observed among infants with pulmonary involvement.

The clinical course of LCH is variable. Patients with unifocal disease generally have an excellent prognosis. After initial bone scanning and radiographic survey to assess the extent of the disease, follow-up studies after treatment should be performed at 6-month intervals for 3 years. If no additional lesions are present at 1 year, the development of subsequent lesions is unlikely. A full recovery is also expected in cases of solitary lymph node involvement or isolated skin disease. In pulmonary LCH, prognosis is more unpredictable. Remission may occur following smoking cessation, while others progress to chronic lung disease. [29, 30] Owing to its unpredictable nature, it is recommended for patients with pulmonary LCH to undergo follow up at least twice a year.

Multifocal LCH has a variable prognosis, especially in patients at the extremes of age with pulmonary involvement. The prognosis is worse than in patients with unifocal disease but better than those with disseminated disease. Sixty percent of patients with multifocal disease have a chronic course, 30% of patients undergo complete remission, and 10% of patients with multifocal LCH die from the disease. [31] Response to chemotherapy in the first 6 weeks (induction therapy) is among the most important prognostic indicators for multifocal LCH. A good response to chemotherapy during this period is associated with significantly improved survival. [4, 32] Conversely, hematologic involvement or involvement of organs such as the lungs, spleen, and liver is associated with worse long-term outcomes. [4]

Studies found that children with V600E BRAF mutations are associated with disease relapse and a worse prognosis. [33] BRAF V600E‒positive mutations were also more likely present in systemic LCH, while patients without the mutation had diseases limited to the skin. [34, 35] However, it is important to note that those studies treated patients with conventional therapy with vinblastine and prednisone. Direct therapies targeting BRAF V600E mutations (vemurafenib) have shown improvements in refractory BRAF V600E‒positive patients, although relapses are still common. [36]

Letterer-Siwe disease (disseminated) has a high mortality rate. The prognosis in these patients depends on the patient's age, the extent of disease, and the degree of organ dysfunction. The mortality rate is 50% or higher.

The congenital form of histiocytosis tends to resolve spontaneously within weeks to months. [37] Although the absence of systemic disease at presentation and the tendency of resolution of the disease are favorable, long-term follow-up care to detect evidence of relapse or progression in these patients is suggested. [37] Relapse in these patients has been reported up to 5 years after the initial disappearance of the disease. Cutaneous lesions usually disappear by 3 months, leaving residual hypopigmentation.

Infrequently, cases originally diagnosed as chronic focal LCH may progress to multifocal or even disseminated disease.


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